Regulatory Environment and Approvals in Cell and Gene Therapy Products Between Japan, the USA, and the EU.

Abstract

This study aimed to demonstrate the differences in the way cell and gene therapy (CGT) products have been developed and reviewed for approval in Japan, the USA, and the EU by comparing regulations and successfully launched products in each region, and to examine the background to such differences. Information on relevant regulations and approved CGT products were collected from the public source and compared by region. While regulations on CGT products are largely consistent among these regions, some differences could have a substantial impact on the practices defining CGT products, the timing of responses required to comply with the regulations for handling gene-modified organisms, and the acceptable validation processes under good manufacturing practice regulations. Although CGT products are given some preferential status in all regions, the preferential treatment given to CGT products varies across regions. The CGT products launched in each region also differ significantly in type, indications, the nature of the developers, and the clinical evidence submitted. While all the cellular products launched in Japan were approved based on small uncontrolled trials, most cellular products in the USA and EU were approved based on controlled studies. A trend was observed for companies to enter their home markets. Our study showed differences of regulations on CGT products and of features in approved products as well as the trend of their home market entries, which may have been driven by a different context than that of traditional pharmaceuticals.